Cystic Fibrosis (CF) is most commonly associated with its effects on the lungs those afflicted. In the lungs, the improperly functioning cystic fibrosis transmembrane conductance regulator (CFTR), which normally transports chloride ions across the lung membrane, results in increased mucous production, poor mucous clearance and a greater risk for contracting severe lung infections, which can ultimately cause death of the patient. Cystic fibrosis (CF) is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR). The pancreas is universally involved in CF with progression to pancreatic insufficiency in most cases within the first few years of life. Pancreatic insufficiency correlates with the severity of lung disease and CF-related diabetes (CFRD), therefore preserving pancreatic function in CF may have an important impact on disease morbidity and mortality. Pancreatic disease is universal in humans and pigs with cystic fibrosis (CF) and progresses to pancreatic insufficiency (PI) in a few years. CF is the most common form of PI in children.
Currently, there are no treatments to prevent the pancreatic disease progression in CF patients. Further, there are no gene therapy trials targeting the pancreas in CF or other pancreatic diseases. Moreover, in animal models, gene therapy techniques for pancreatic diseases suffer from the lack of effective and safe delivery methods.